Fabhalta® (iptacopan capsules), first oral treatment for adult patients with PNH, now available in Canada

• Fabhalta is the first oral monotherapy for previously treated and treatment-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH),^1,2 a chronic, rare and potentially life-threatening blood disorder ^3,4
• Now available in Canada, this new treatment option helps address significant unmet needs for adult patients with PNH; a large proportion of patients can remain anemic and dependent on blood transfusions^3,4
• Canada's Drug Agency (CDA) and Institut National d'Excellence en Santé et Services Sociaux (INESSS) have recommended reimbursement of Fabhalta in the treatment of adult patients with PNH who have an inadequate response to, or are intolerant of, a C5 inhibitor^5,6
• In clinical trials, treatment with Fabhalta offered superior hemoglobin improvement in the absence of red blood cell (RBC) transfusions^1,7,8,9,10

MONTREAL, April 14, 2025 /CNW/ - Novartis Canada is pleased to announce that Fabhalta^® (iptacopan capsules) is now available across Canada for adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia.¹ The first and only oral monotherapy treatment indicated for PNH, Fabhalta received Health Canada approval in January 2025.

"I received my PNH diagnosis over 20 years ago, when knowledge of the disorder was limited and there were no treatments available. It is exciting to experience how the treatment landscape is evolving and improving for the PNH community," said Barry Katsof, President, Canadian Association of PNH Patients. "The availability of a new oral treatment option will provide meaningful benefits for Canadians who continue to struggle with anemia and dependence on blood transfusions, despite their current therapies."

Complement Component C5 is a gene that encodes a component of the complement system, a part of the body's immune system that helps defend the body against injury and foreign invaders, like bacteria and viruses.^11,12 Existing C5 inhibitor treatments, administered as infusions, may leave PNH symptoms uncontrolled.^3,4 In fact, patients on anti-C5 treatment  have reported persistent anemia, with some patients requiring blood transfusions at least once per year.^3,4 Fabhalta is the only Factor B inhibitor – a small-molecule therapy that treats diseases caused by dysregulation of the alternative complement pathway – providing comprehensive control of RBC destruction within and outside the blood vessels (intra- and extravascular hemolysis [IVH and EVH]) approved in Canada.

"In clinical trials, iptacopan was superior to C5 inhibition in terms of increasing hemoglobin and achievement of transfusion-avoidance in treatment-experienced patients. It was also shown to be effective in improving hemoglobin responses without the need for transfusions in complement inhibitor-naive patients. In both studies, LDH values were well-controlled, suggesting inhibition of terminal complement function," ^7,13 said Dr. Christopher Patriquin, Assistant Professor of Medicine and Clinician Investigator, University of Toronto. "The availability of a safe and effective oral treatment for PNH^7,8,13,14 offers physicians more flexibility in personalizing care for patients, potentially easing limitations faced by those receiving injectable therapies."

"We remain committed to innovation that improves the lives of Canadians with PNH and are proud to bring a new oral medicine to those living with this chronic and life-altering blood disease," said Mark Vineis, Country President, Novartis Canada. "As the first and only oral treatment for PNH, we are confident the availability of this new treatment option will support improved outcomes while providing renewed hope and freedom for patients, their loved ones and the healthcare providers who care for them."

Novartis is proud to offer a Patient Support Program (PSP), Fabhalta Assist.

The clinical effectiveness, safety and cost-effectiveness of Fabhalta in PNH has been reviewed by Canada's Drug Agency (CDA) and Institut National d'Excellence en Santé et Services Sociaux (INESSS). Both have issued a positive recommendation in favor of the reimbursement of Fabhalta in the treatment of adult patients with PNH who have an inadequate response to, or are intolerant of, a C5 inhibitor.^5,6 Novartis looks forward to working with provinces so that eligible PNH patients can access this new option.

About paroxysmal nocturnal hemoglobinuria (PNH)
PNH is a rare, chronic and potentially life-threatening complement-mediated blood disorder² (a disease that occurs when the complement system is activated inappropriately or in excess). People with PNH have an acquired mutation in some of their hematopoietic stem cells (which are located in the bone marrow and can grow and develop into RBCs, white blood cells and platelets) that causes them to produce RBCs that are susceptible to premature destruction by the complement system.^2,4 This leads to intravascular hemolysis (destruction of RBCs within blood vessels) and extravascular hemolysis (destruction of RBCs mostly in the spleen and liver), which cause anemia (low levels of circulating RBCs), thrombosis (formation of blood clots) and other debilitating symptoms in varying combinations and levels of severity.^2-4

It is estimated that approximately 10-20 people per million worldwide live with PNH.² Although PNH can develop at any age, it is often diagnosed in people between 30-40 years old.^15,16

About Fabhalta (iptacopan capsules) 
Fabhalta is an oral, Factor B inhibitor of the alternative complement pathway.^17,18,19 Fabhalta is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia. 

Fabhalta is only available through a controlled distribution program under which prescribers must enroll patients and confirm vaccination against encapsulated bacteria.¹

Health Canada approval was based on the Phase III APPLY-PNH trial in patients with residual anemia despite prior anti-C5 treatment who switched to Fabhalta^1,7 and supported by the Phase III APPOINT-PNH study in complement inhibitor-naïve patients.^1,8 

About Novartis 
Novartis is a focused innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach more than 250 million people worldwide.

In Canada, Novartis Pharmaceuticals Canada Inc. employs approximately 600 people to serve the evolving needs of patients and the healthcare system and invests over $30 million in R&D yearly in the country. For more information visit www.novartis.ca.

References

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¹ Fabhalta (iptacopan capsules) Product Monograph. https://www.novartis.com/ca-en/sites/novartis_ca/files/fabhalta_scrip_e.pdf. January 6, 2025.

² Cançado RD, Araújo A da S, Sandes AF, et al. Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria. Hematol Transfus Cell Ther. 2021;43(3):341-348. doi:10.1016/j.htct.2020.06.006

³ McKinley CE, Richards SJ, Munir T, et al. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130(Supplement 1):3471. doi:10.1182/blood.V130.Suppl_1.3471.3471

⁴ Dingli D, Matos JE, Lehrhaupt K, et al. The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey. Ann Hematol. 2022;101(2):251-263. doi:10.1007/s00277-021-04715-5

⁵ Reimbursement Recommendation: Iptacopan (Fabhalta). Canadian Journal of Health Technologies. Canada's Drug Agency. https://www.cda-amc.ca/sites/default/files/DRR/2025/SR0851_Fabhalta_FINAL_Recommendation.pdf. Accessed on April 7, 2025.

⁶ Extract Notice to the Minister: Fabhalta (HPN). INESSS. https://www.inesss.qc.ca/en/themes/medicaments/drug-products-undergoing-evaluation-and-evaluated/extract-notice-to-the-minister/fabhalta-hpn-7233.html. Accessed on April 7, 2025.

⁷ Risitano AM, Rӧth A, Kulasekararaj A, et al. Oral Iptacopan Monotherapy Has Superior Efficacy to Anti-C5 Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria and Residual Anemia: Results From the Phase III APPLY-PNH Study. Presented at: 49th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT); April 23-36, 2023; Paris, France.

⁸ Risitano AM, Han B, Ueda Y, et al. Oral Complement Factor B Inhibitor Iptacopan Monotherapy Improves Hemoglobin to Normal/Near-Normal Levels in Paroxysmal Nocturnal Hemoglobinuria Patients Naïve to Complement Inhibitors: Phase III APPOINT-PNH Trial. Presented at: 49th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT); April 23-36, 2023; Paris, France.

⁹ Risitano AM, Röth A, Soret J, et al. Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single-arm, phase 2, proof-of-concept trial. Lancet Haematol. 2021;8(5):e344-e354. doi:10.1016/S2352-3026(21)00028-4

¹⁰ Jang JH, Wong L, Ko BS, et al. Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study. Blood Adv. 2022;6(15):4450-4460. doi:10.1182/bloodadvances.2022006960

¹¹ C5 complement C5 [ Homo sapiens (human) ]. Gene ID: 727. National Library of Medicine. https://www.ncbi.nlm.nih.gov/gene/727. Accessed on January 8, 2025.

¹² Complement System. Cleveland Clinic. https://my.clevelandclinic.org/health/body/23370-complement-system. Accessed April 1, 2025.

¹³ Novartis Pharmaceuticals. A Randomized, Multicenter, Active-Comparator Controlled, Open-Label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult Patients With PNH and Residual Anemia, Despite Treatment With an Intravenous Anti-C5 Antibody. clinicaltrials.gov; 2022. Accessed September 21, 2022. https://clinicaltrials.gov/ct2/show/NCT04558918

¹⁴ Novartis Pharmaceuticals. A Multicenter, Single-Arm, Open-Label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy. clinicaltrials.gov; 2022. Accessed September 21, 2022. https://clinicaltrials.gov/ct2/show/NCT04820530

¹⁵ Hill A, DeZern AE, Kinoshita T, Brodsky RA. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primer. 2017;3(1):17028. doi:10.1038/nrdp.2017.28

¹⁶ Schrezenmeier H, Röth A, Araten DJ, et al. Baseline clinical characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry. Ann Hematol. 2020;99(7):1505-1514. doi:10.1007/s00277-020-04052-z

¹⁷ Schubart A, Anderson K, Mainolfi N, et al. Small-molecule factor B inhibitor for the treatment of complement-mediated diseases. Proc Natl Acad Sci. 2019;116(16):7926-7931. doi:10.1073/pnas.1820892116

¹⁸ Barratt J, Rovin B, Zhang H, et al. POS-546 EFFICACY AND SAFETY OF IPTACOPAN IN IgA NEPHROPATHY: RESULTS OF A RANDOMIZED DOUBLE-BLIND PLACEBO-CONTROLLED PHASE 2 STUDY AT 6 MONTHS. Kidney Int Rep. 2022;7(2):S236. doi:10.1016/j.ekir.2022.01.577

¹⁹ Rizk DV, Rovin BH, Zhang H, et al. Targeting the Alternative Complement Pathway With Iptacopan to Treat IgA Nephropathy: Design and Rationale of the APPLAUSE-IgAN Study. Kidney Int Rep. 2023;8(5):968-979. doi:10.1016/j.ekir.2023.01.041

SOURCE Novartis Pharmaceuticals Canada Inc.

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